Chronic kidney disease (CKD) in children involves a host of rare diseases affecting children of all ages. The therapeutic needs of these children are largely unmet because of limited disease- and age-specific drug development. The absence of pediatric testing to document appropriate pediatric dosing, safety, and efficacy has many consequences:
■ Few drugs are labeled for use in children with CKD.
■ Off-label prescribing is often extrapolated from product labels written for adult patients with kidney disease or other non–kidney-related conditions.
■ Little progress has been made to fill the information gaps required to guide the pharmacologic treatment of children with kidney diseases.
Drug development for children with kidney diseases must consider issues unique to this vulnerable population. Legislation by the United States and the European Union mandates plans for pediatric development as part of an overall product development strategy. This highlights the need to prioritize those programs that may be deemed most necessary and impactful and to optimize designs to facilitate their successful completion.
The Kidney Health Initiative (KHI) is engaging with relevant stakeholders, including patients, healthcare providers, researchers, professional organizations, industry partners, and regulators, to address these issues by developing recommendations to foster drug development for children with kidney diseases.